Cell-Based Therapies: Transitioning from the Research Laboratory to the Clinic

Cell therapies have been used in clinical trials for more than 25 years, but until recently, their effectiveness and clinical application has been limited. The NIH intramural program is well suited to develop and translate novel cell therapies in early phase clinical trials. The results of many of these trials have been promising and some have advanced to later phase clinical trials. This symposium will highlight advances being made by intramural investigators in the fields of cancer immunotherapy, gene therapy, and regenerative medicine and the unique role of the Clinical Center and intramural program in the rapidly emerging field of cell-based therapies.

Developmental Biology

Developmental biology aims to understand how an organism develops—how a single cell becomes an organized grouping of cells that is then programmed at specific times to become specialized for certain tasks. Genes control much of an organism’s development, but environmental stimuli also play a role, resulting in the complex “nature vs. nurture” paradigm. Intramural Research Program (IRP) investigators seek a deeper understanding of human development and the many disorders and diseases that can occur when normal development is disrupted. The purpose of this symposium is to inform the NIH community of the breadth of research in Developmental Biology being carried at the NIH in different laboratories, using a variety of animal species to model human biology.

Genotyping and phenotyping

The genomics era has led to exciting discoveries in human clinical genetics that have greatly improved our understanding of the etiology of rare and common diseases. This symposium will highlight studies incorporating clinical characterization at the NIH Clinical Center with discovery of their genetic etiology. The topics range of topics discussed this symposium including neuromuscular disease, cancer predisposition syndromes, and immunodeficiency are meant to illustrate the breadth and depth of such studies in the intramural research program and highlight opportunities for collaboration.

Neuroscience and compulsive disorders

Hunger-driven motivational state completionMichael Krashes, Ph.D. (NIDDK)Goal-directed behaviors are sets of motor actions that direct an animal toward an explicit target object, an interaction that promotes individual survival and/or maintains the species. Despite the necessity of motivated behaviors ranging from ingestive to reproductive to aggressive/defensive exploits, an organism can only perform a single action at any given time, highlighting the tremendous flexibility and speed in which the brain can coordinate complex decision-making. Here Dr. Krashes’s team investigates the role distinct levels of satiety play on shaping innate, motivated drive states as well as the subsets of neurons capable of perturbing innate behavioral choice.

Alcohol drinking: what is driving the higher risk for alcohol abuse?Veronica A. Alvarez, Ph.D. (NIAAA)Several factors have been associated with high alcohol drinking in humans. The team lead by Dr. Alvarez is investigating whether similar factors determine vulnerabilities to high alcohol use in rodents and whether there is link and common mechanism underlying these vulnerabilities.

Role of anterior insular cortex and amygdala circuits in relapse after voluntary abstinenceYavin Shaham, Ph.D. (NIDA)Dr. Shaham will describe a new rat model of drug relapse after choice-based voluntary abstinence and present recent findings on the role of D1- and D2-receptor expressing neuronal ensembles in dorsomedial striatum and glutamatergic projections from anterior insular cortex to central amygdala in this new form of relapse.

Compulsive and impulsive disorders: polar opposites?Philip Shaw, Ph.D. (NHGRI)Dr. Shaw will consider the insights that can be gained into compulsivity through considering the neural substrates of childhood obsessive compulsive disorder. The focus will be on contrasts that can be drawn with childhood disorders in which impulsivity is a prominent symptom- such as attention deficit hyperactivity disorder.

Diversity of biological insights from single cell analysis across multiple diseases

This symposium will highlight the application of single cell genomics across many different areas of biomedical interest. We will discuss a variety of exciting cellular and genomics techniques used as well as the challenging but informative informatics analyses that can be applied in this area. We hope that the audience will see opportunities to apply single cell techniques to their own research questions.

RNA biology and therapeutics

DNA serves as the repository for genetic information, and proteins are the biochemical machines of the cell. RNA is remarkable, being able to carry out both functions. This biochemical and structural versatility gives rise to diverse cell biological roles ranging from catalysis to the regulation of chromatin structure and gene expression. Because of its many critical functions, RNA has emerged as an important target for therapeutic intervention. In this session, advances in fundamental RNA biology, structure-based engineering, and pharmacological targeting of RNA in the NIH intramural program will be presented.